Tisagenlecleucel

T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor that reacts to cancer, and transferred back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells . A chimeric T cell receptor (” CAR-T “) is expressed on the surface of the cell.

It was invented and initially developed at the University of Pennsylvania; Novartis completed development, obtained FDA approval, and markets treatment. [1] In August 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States. [2]

It is administered in a single treatment, which will cost $ 475,000. Novartis says this is cheaper than some bone marrow transplants. Novartis says it will not charge people who do not respond. [3]

History

The treatment was developed by Carl H. June at the University of Pennsylvania and is licensed to Novartis. [4]

In April 2017, CTL019 received breakthrough therapy designation by the US FDA for the treatment of relapsed or refractory broad-based B-cell lymphoma . [5]

In August 2017 the FDA grants approval for the use of tisagenlecleucel in people with acute lymphoblastic leukemia. [9] According to Novartis, the treatment will be given to specific medical centers where these patients have been trained to respond to this type of treatment. [10]

Manufacture

In a 22-day process, the treatment is customized for each person. T cells are purified from blood cells, which are then modified by a virus that inserts a gene into the cells’ genome . The gene encodes a chimaeric antigen receptor(CAR) that targets leukemia cells. [6]

It uses the 4-1BB co-stimulatory domain in its CAR to improve response. [11]